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US 11,642,384 B2Granted
Compositions and methods for the treatment of Netherton Syndrome
AssigneeKrystal Biotech, Inc.
InventorsPooja Agarwal, Suma Krishnan, John C. Freedman
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Abstract
The present disclosure provides recombinant nucleic acids comprising one or more polynucleotides encoding a Serine Protease Inhibitor Kazal-type (SPINK) polypeptide (e.g., a SPINK5 polypeptide); viruses comprising the recombinant nucleic acids; compositions and formulations comprising the recombinant nucleic acids and/or viruses; methods of their use (e.g., for the treatment of Netherton Syndrome); and articles of manufacture or kits thereof.
Claims7
A pharmaceutical composition comprising:
- (a) a replication defective herpes simplex virus comprising a recombinant herpes simplex virus genome, wherein the recombinant herpes simplex virus genome comprises one or more polynucleotides encoding a Serine Protease Inhibitor Kazal-type 5 (SPINK5) polypeptide;
- and
- (b) a pharmaceutically acceptable excipient,
- wherein the one or more polynucleotides encoding the SPINK5 polypeptide are operably linked to a promoter suitable for transcription in a mammalian cell.
1 more element
Dependencies are inferred from the claim text.